Shots:
Samsara Biocapital is a California-based venture capital and registered investment adviser investing across early to post-IPO stages in healthcare, life sciences, oncology, and digital health, with a portfolio of 70+ companies including Scholar Rock and PepGen
The firm made 30 investments across PIPE (43%), Series B (23%), and other rounds, with notable deals such…
Shots:
The US FDA received BLA, plus a request for a priority review of apitegromab to improve motor function in pts with SMA who are on SMN-targeted therapy. The EMA’s MAA to be filed in Q1’25
The BLA was supported by the P-III (SAPPHIRE) & P-II (TOPAZ) trial, where SAPPHIRE met its 1EP of…
Shots:
Dr. Jay Backstrom in an engaging conversation with PharmaShots, shares the highlights of the P-II Topaz trial designed to evaluate the safety and efficacy of apitegromab for the treatment of Spinal Muscle Atrophy (Type 2 & 3)
With the potential to transform care in SMA by directly addressing progressive muscle weakness, apitegromab aims to…
Shots:
Nagesh talked about how Spinal Muscular Atrophy (SMA) is affecting people all over the world and how Scholar Rock’s treatment options are improving the lives of those living with this disease
Nagesh also spoke about Scholar Rock’s lead product in SMA, apitegromab, highlighting its trial designs (TOPAZ & SAPPHIRE trials), outcomes, and real-world data…

